To improve the understanding of clinicians on the diagnostic criteria and treatment principlis of pulmonary alveolar proteinosis (PAP),which is a rare respiratory disease. European Respiratory Society published the first edition guidelines for PAP, including a systematic review of the literature and the application of the grading of recommendations, assessment, development and evaluation (GRADE) approach to assess the certainty of evidence and the strength of recommendations. Five questions of patient, intervention, comparison, outcome (PICO) and two narrative questions were developed. Recommendations and evidence-based evidence were given, including management of PAP, whole lung lavage, granulocyte-macrophage colony-stimulating factor (GM-CSF) therapy, rituximab, plasma exchange, and lung transplantation. In addition, recommendations were given for the use of GM-CSF antibody testing, bronchoalveolar lavage, and lung biopsy. This study is to interpret the main content of the guideline.

Objective To investigate the intervention effects and mechanism of icariin on prostate cancer based on network pharmacology and animal experiments. Methods The targets of icariin were predicted using the SwissTargetPrediction database. Protein-protein interaction networks were constructed with the String database, and core targets were screened using Cytoscape 3.9.1. GO and KEGG enrichment analysis on core targets were conducted with the Metascape database to predict the mechanism of action. A PC-3 tumor-bearing mouse model of prostate cancer was established to observe the inhibitory effects of icariin alone and in combination with paclitaxel on tumor growth. Results Network pharmacology predictions suggested that icariin has potential therapeutic effects on prostate cancer, with core targets potentially including serine/threonine kinase 1 (AKT1), B-cell lymphoma-2 (BCL2), epidermal growth factor receptor (EGFR), heat shock protein 90 alpha family class A member 1 (HSP90AA1), heat shock protein 90 alpha family class B member 1 (HSP90AB1), nuclear factor kappa B subunit 1 (NF-κB1), tumor protein p53, etc. Animal experiments found that compared with the model control group, the tumor volume growth in the icariin group and the paclitaxel group was significantly inhibited, and the serum tumor necrosis factor content was significantly reduced, while testosterone levels did not change significantly. Both groups significantly downregulated the mRNA expression of Notch1, Jagged1 and Hes1(P<0.05), with the combined treatment group showing a more significant inhibitory effect. Conclusions Both network pharmacology and animal experimental results confirmed that icariin has a significant inhibitory effect on prostate cancer. One of the mechanisms of its anti-tumor effects may be the significant inhibition of the activated Notch signaling pathway in tumors.

Objective To identify the main blood absorbed constituents from Youguiyin and elucidate the proliferative effect of Youguiyin and its main blood absorbed constituents on osteoblasts in rats. Methods Ultra-performance liquid chromatography-mass spectrometry (UPLC-MS) technique was used to establish the analysis method for Youguiyin, individual herbal medicines and the medicated serum containing Youguiyin after oral administration in Sprague-Dawley rats. Osteoblasts of rats were isolated and cultured in vitro. The effect of Youguiyin, medicated serum, and its main blood absorbed components on osteoblast proliferation rate was evaluated by thiazole blue (MTT) assay. Results Ten components were detected in the blood serum of rats after oral administration of Youguiyin, namely aconitine, hypaconitine, mesaconitine, benzoylaconitine, benzoylhypaconitine, benzoylmesaconine from Aconiti Lateralis Radix Praeparata, cinnamic acid from Cinnamomi Cortex, morroniside, loganin from Corni Fructus, and pinoresinoldiglucoside from Eucommiae Cortex, respectively. Youguiyin decoction and seven components from Aconiti Lateralis Radix Praeparata and Cinnamomi Cortex showed a certain promoting proliferation effect on osteoblasts when the concentrations were 1 μg·mL^-1,10 ng·mL^-1,10 ng·mL^-1,1 ng·mL^-1,100 μg·mL^-1,100 pg·mL^-1,1 μg·mL^-1 and 1 ng·mL^-1, respectively. Besides, the effect onset was relatively rapid. The onset time of medicated serum containing Youguiyin was slow. Conclusions The ten chemical components absorbed in the blood from Youguiyin were identified. It was revealed that Youguiyin, medicated serum and the main components of Youguiyin absorbed in the blood can promote the proliferation of osteoblasts within a certain concentration range.

Objective To reveal the pharmacodynamic material basis of Kaixinsan by using high performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) and the integrated analysis of "chemical component spectrum-plasma exposure component spectrum-mitochondrial function". Methods Through a review of literature, databases, and previous studies, the chemical components of ginseng, polygala, poria, and acorus were systematically cataloged. A qualitative analysis method for the chemical constituents in the aqueous extract of Kaixinsan was developed, allowing for the identification of its chemical components. A qualitative analysis for rat plasma based on HPLC-MS/MS was established, which was applied to analyze the plasma exposure component spectrum following oral administration of Kaixinsan aqueous extract in rats. Aerobic respiration was evaluated using a seahorse cell energy metabolism analyzer, and the effect of key components of Kaixinsan on mitochondrial aerobic respiration was assessed. Results Four main types of components were identified in the Kaixinsan aqueous extract, including saponins, oligosaccharide esters, xanthones, and triterpenes, comprising a total of 231 identified compounds. Analysis of rat plasma 30 minutes after gavage with Kaixinsan identified 55 compounds. The analysis revealed that ginsenoside Rg1, 3,6'-disinapoylsucrose, polygalaxanthone III and poricoic acid B could significantly enhance mitochondrial respiratory capacity using in vitro cellular assays to detect aerobic respiration of four main components entered blood. Conclusions Saponins, oligosaccharide esters, xanthones, and triterpenes may be the material basis for the pharmacological effect of Kaixinsan by improving mitochondrial function. The integrated analysis of "chemical component spectrum-plasma exposure component spectrum-mitochondrial function" provides a new approach for in-depth exploration of the material basis underlying the efficacy of traditional Chinese medicine.

Objective To investigate the mechanism of Guizhi Tongluo Tablets (GZTLP) on improving atherosclerosis in APOE knockout mice by regulating neutrophil extracellular trapping nets (NETs). Methods After modeling, 24 APOE knockout mice aged 8 weeks were randomly divided into 4 groups: GZTLP high-dose group, low-dose group, model control group and normal control group, with 6 mice in each group. GZTLP was given 1.87 mg·g^-1 and 0.47 mg·g^-1 intragastric administration in high-dose group and low-dose group, respectively. The normal control group and model control group were given 0.9% sodium chloride solution intragastric administration for 6 weeks, and the lipid plaque deposition in aorta was observed by gross oil red O staining. Lipid deposition in aortic root was observed by oil red O staining. The pathological changes of lipid plaques in aortic root were observed by HE staining. The levels of interleukin-1β (IL-1β) and tumor necrosis factor α (TNF-α) in peripheral blood of mice were detected by enzyme-linked immunosorbent assay (ELISA). The expression of lymphocyte antigen 6G (Ly6G), myeloperoxidase (MPO) and citrulinated histone (Cit-H3) in plaques of the aortic arch and the colocalization of Ly6G, MPO and Cit-H3 were detected by immunofluorescence assay. Results Compared with the normal control group, the aorta of mice in the model control group showed serious lipid plaque deposition, morphological damage, and a large number of inflammatory cells infiltration, the contents of serum inflammatory factors IL-1β and TNF-α were increased, and the protein expressions of Ly6G, Cit-H3 and MPO were significantly increased. Compared with model control group, GZTLP group reduced the amount of lipid plaque deposition in aorta, the arrangement of aortic cells was more regular, the inflammatory cell infiltration was improved, and the contents of serum inflammatory factors IL-1β and TNF-α were significantly decreased (P<0.05). The colocalization and the protein expression of Ly6G, MPO and Cit-H3 were significantly decreased in aortic tissues (P<0.01). Conclusions GZTLP can improve atherosclerosis, and its mechanism may be related to the inhibition of neutrophil extracellular trapping nets.

Objective To investigate the repairing effect and mechanism of Chinese patent medicine Weiyangning pill on gastric mucosal injury in rats induced by anhydrous ethanol, and to establish a high-performance liquid chromatography (HPLC) method to determine the five main components of Weiyangning pill. Methods The five components of paeoniflorin, psoralen, atractylenolide III, liquiritin and hesperidin in Weiyangning pill were detected by HPLC. SD male rats were randomly divided into normal control group, model control group, Weinaian group, and large and small dose group of Weiyangning pill. All rats were fasted for 24 hours without water fasting. The normal control group and the model control group were given purified water by gavage. While Weinaian group was given Weinaian (3 g·kg^-1), the test group were given intragastric perfusion of Weiyangning (3,1.5 g·kg^-1) respectively. After 2 hours, all the rats, except the normal control group, were intragastrically administered with anhydrous ethanol (5 mL·kg^-1) to establish the model of gastric mucosal injury. An hour later, the experimental materials were collected, and the gross score of gastric mucosal injury was observed and calculated. The gastric mucosal slices were stained by hematoxylin-eosin (HE) to calculate the pathological scores. Immunohistochemistry was employed to detect the expression of gastric mucosa-related proteins. Results The high-performance liquid chromatogram of Weiyangning pill was obtained, and the absorption peaks with the same retention time as the five standard substances (paeoniflorin, psoralen, atractylenolide III, liquiritin and hesperidin) were observed. The general score and pathological score of gastric mucosal injury in Weiyangning groups (3,1.5 g·kg^-1) were lower than those of the model control group ( P<0.05 or P<0.01 ). Weiyangning pill (3,1.5 g·kg^-1) ameliorated the decrease expression of tight junction protein (Claudin-7), adhesion junction proteins (E-cadherin and β-catenin), mucins (MUC1 and MUC5AC), and the gastric transcription factor SOX2 in the gastric mucosa of the rats modeled in anhydrous ethanol (P<0.05 or P<0.01 compared with the model control group). Conclusion The repairing effect of Weiyangning pill on gastric mucosal injury induced by anhydrous ethanol in rats is related to the increase of the expression of tight junction protein, adhesion junction protein, mucin and gastric transcription factor.

Objective To establish a drosophila reproductive dysfunction model and observe whether Danggui Shaoyao San (DSS) has an improving effect. Methods Tripterygium glycosides were used to establish the drosophila reproductive dysfunction model. The number of offspring pupae, the parental superoxide dismutase (SOD) and catalase (CAT) activities, the parental reproductive-related gene expression, the F1 generation body weight, and the F1 generation development-related genes were measured to evaluate the efficacy of DSS. Results Tripterygium glycosides in a dose of 20 mg·mL^-1 significantly inhibited the quantity of offspring ( P<0.01) and were suitable for establishing the drosophila reproductive dysfunction model. Compared with the model control group, medium (10 mg·mL^-1) and high (20 mg·mL^-1) dose DSS significantly increased the number of offspring pupae (P<0.01, P<0.05). High-dose DSS significantly increased the SOD and CAT activities of the parent drosophila (P<0.01). Medium and high doses of DSS promoted the expression of drosophila parental reproduction-related genes, among which the medium dose DSS increased the mRNA expression of DEAD-Box helicase 4(VASA) and factor in the germline alpha (Figla) (P<0.05, P<0.01), and the high dose DSS increased the mRNA expression of VASA, Figla and forkhead box O (FoxO) (P<0.01). The medium dose of DSS significantly increased the body weight of F1-generation female drosophila (P<0.05), and the high dose of DSS significantly increased the body weight of both F1-generation female and male drosophila (P<0.05, P<0.01). Both the medium and high dose DSS increased the mRNA expression of the F1 generation development-related gene mammalian target protein of rapamycin (mTOR) (P<0.01). Conclusions DSS improves the reproductive function injury of drosophila induced by tripterygium glycosides.

Objective This study systematically evaluated the clinical efficacy of Shangke Jiegu tablet in the treatment of fracture, and explored the mechanism of action of Shangke Jiegu tablet and the compatibility of each efficacy group from the“Disease-Symptom-Formula”perspective. Methods Clinical research literatures on the use of Shangke Jiegu tablet for fracture intervention were retrieved from Chinese databases (CNKI, Wanfang Database, VIP database) and English databases (PubMed, Cochrane Library, EMbase), covering the period from the inception of the databases to January 2024. Risk assessment tools were used to evaluate the literature’s quality, and the data were extracted and analyzed using Stata 16.0 software. Gene sets associated with fracture symptoms were identified through the TCMIP platform (version 2.0). Differential gene expression related to fractures was obtained from the GEO database. Chemical composition and candidate target profiles of the 12 herbs in Shangke Jiegu tablets were collected from TCMIP v2.0. An interaction network between fracture-related genes and drug candidate targets was established, and core network targets were screened based on topological features, with functional enrichment analysis performed. Results A total of 14 articles were incorporated into the Meta-analysis, encompassing a total sample size of 1 293 cases, indicating an overall response rate of Shangke Jiegu tablets in fracture therapy (RR=1.24, 95% CI: 1.18-1.31, P<0.001). The "Disease-Symptom-Formula" association network analysis indicated that the pathways related to the putative targets of Shangke Jiegu tablet were primarily involved in bone healing, nerve and blood system regulation, and immune-inflammation regulation. Different efficacy groups within the prescriptions showed varying emphases on these roles. Conclusions Shangke Jiegu tablet may facilitate fracture healing by regulating blood and nervous systems, correcting immune-inflammatory imbalances, and maintaining bone and energy metabolism. The comprehensive effects include the dissipation of blood stasis, the promotion of blood circulation, the alleviation of swelling and pain, the regeneration of muscles and bones, and the clearance of heat and detoxification. These findings support the clinical advantages and positioning of Shangke Jiegu tablet.

Objective To analyze the current state of medication literacy among elderly patients with chronic diseases in urban areas of Beijing, identify its key influencing factors, and propose targeted improvement measures. Methods From February 1 to June 30, 2023, a questionnaire survey was conducted among elderly chronic disease patients in 193 communities across 15 districts of Beijing using a convenience sampling method. Data was collected through face-to-face interviews, with a total of 787 questionnaires distributed and 755 valid responses received.The logistic regression analysis model was employed to systematically identify and evaluate the factors affecting patients' medication literacy. Results Among the 755 valid questionnaires collected, 53.25% (402 cases) of patients met the medication literacy criteria. Regression analysis results showed that multiple factors significantly influenced medication literacy among elderly patients with chronic diseases, including using rural cooperative medical care or self-payment methods (OR=1.669, P=0.039), retaining medication instructions (OR=0.519, P=0.038), checking medication instructions before use (OR=1.993, P<0.01), and possessing the ability to understand medication instructions (partial understanding OR=2.805, P=0.038; fully understanding OR=3.084, P=0.022) as positive influencing factors; whereas having 2 to 3 chronic diseases (OR=0.574, P=0.039), taking 3 to 5 medications (OR=1.845, P=0.015), and experiencing drug-related problems (OR=1.993, P<0.01) were identified as negative influencing factors. Conclusion Multiple factors influence medication literacy among elderly patients with chronic diseases. To ensure the safety and efficacy of their medication use, It is recommended to implement targeted measures. These include revising patient medication guidance leaflets tailored to age, enhancing patients' understanding of drug instructions, and strengthening medication guidance and social support systems.

Objective To analyze the differences in aging-related information between Chinese and American drug instruction for common chronic disease in the elderly, and to propose policy recommendations for the aging-related drug instruction in China. Methods Ten common chronic disease treatment drugs for elderly patients were selected, and through the random sampling method, the drug manuals of one domestic manufacturer were randomly selected for each drug by the random number method, and one American drug manual was selected as a sample from the dailymed website, and the information related to elderly people in the various items of the drug manuals was statistically analyzed by using Excel for comparative analysis. The average reading level of the patient version of the U.S. drug instructions was calculated using the Readability Formulas Scoring System; the readability of the Chinese drug instructions was assessed by the Health Education Text Material Suitability Scale. Results Compared with the U.S. drug instruction, the drug instruction for common chronic diseases of the elderly in China were updated less frequently and at a slower pace; the drug instruction with information on medication for elderly patients were fewer than those of the U.S. drug instruction, and the content of the drug instruction was not well documented; and the drug instruction were poorly readable, which was not conducive to the comprehension of the elderly patients. Conclusion China is in urgent need of reforming the aging of drug instructions, and it is recommended that, for chronic disease medications for elderly patients, we should increase the number of clinical trials conducted on the elderly, improve and complete the drug information for the elderly in the drug instructions, and produce "drug instructions for elderly patients" that are suitable for the elderly to read and understand.

Objective To explore the current status of medication literacy among urban elderly patients with chronic diseases in Anhui Province, aiming to reveal the factors influencing their medication literacy, and to propose targeted measures for improvement. Methods This research involved 381 participants aged 60 and above. It was conducted in Anhui province between December, 2022 and January, 2023, with data collected through face-to-face interviews by pharmacists. Single- factor analysis and ordinal multi-class logistic regression analysis were conducted to determine factors affecting medication literacy. Results Medication literacy cognition and medication literacy behavior were rated as good among urban older adults in Anhui province of the 294 valid questionnaires. Those who did not understood package insert exhibited significantly lower medication literacy behavior than those who fully understood [estimate=-1.224, 95% CI=(-2.130,-0.317),P<0.01]. Elderly patients with chronic diseases faced issues such as an inability to read or understand drug instructions in the investigation. 90.48% of elderly patients with chronic diseases never heard or seldom heard of medication guidance services. Conclusion Medication literacy among urban elderly patients with chronic diseases is generally good in Anhui province. The ability to understood drug instructions significantly influenced the medication literacy of urban elderly patients with chronic diseases. Modifying the drug instructions to meet the reading needs of the elderly patients with chronic diseases and developing pharmaceutical care could effectively enhance rational drug use among this demographic.

Objective To evaluate the status of home medication safety among elderly patients with chronic diseases and analyze its influencing factors in urban and rural areas of Henan Province. Methods Convenient sampling method was adopted. Data was collected through a designed and optimized questionnaire. Pharmacists conducted in-home surveys. Excel and SPSS 26.0 software were used for data analysis. Results A total of 352 valid questionnaires were analyzed. Most respondents were aged 60~70 years, with a female proportion of 56.2%, and 52.0% of them had a junior high school education or lower. The top-ranked chronic diseases were hypertension (62.2%) and hyperlipidemia (33.2%), and 61.1% of patients suffered from multiple chronic diseases. Antipyretic and analgesic drugs were most common in home medicine cabinets (90.1%). The proportion of drugs being stored in accordance with the instructions (46.9%) was slightly low, and 93.8% of the elderly handled expired drugs improperly. Awareness of medication guidance services was low (34.9%). Logistic regression analysis showed that factors such as education level and monthly income had significant effects on the understanding of drug package insert, rational drug storage, proper disposal of expired drugs, and awareness of medication guidance services (P<0.05). Conclusion There are still some potential safety risks in the home medication use of elderly patients with chronic diseases in urban and rural areas of Henan Province. It is necessary to enhance education on drug storage and medication guidance, particularly for low-income and less-educated groups, to improve the safety of home medication use.

Objective This study aims to provide an in-depth analysis of the effectiveness of blood pressure control and the influencing factors in elderly hypertensive patients in urban areas of Beijing, providing scientific support for developing more precise and effective home pharmaceutical intervention strategies. Methods Pharmacists conducted home visits and administered questionnaires to systematically investigate elderly hypertensive patients aged 60 and above in the urban areas of Beijing from February to June 2023. Binary logistic regression was used to identify key factors affecting blood pressure control. Results A total of 575 questionnaires were collected with 560 valid responses, achieving an effective recovery rate of 97.39%. Among the respondents, 233 were male (41.61%) and 327 were female (58.39%), with a median age of 69. The proportion of patients with qualified blood pressure control was 33.39% (187 cases). Logistic regression analysis further revealed that smoking, the presence of comorbidities, and multiple comorbid conditions significantly impacted the control rate (P<0.05). Conclusion The blood pressure control rate among elderly hypertensive patients in urban areas of Beijing remains low. It is recommended to develop more targeted home pharmaceutical intervention measures for patients who smoke and have multiple comorbidities to enhance blood pressure control outcomes.

Objective To investigate the application of gene panel in neonates with suspected genetic metabolic diseases. Methods The gene panel designed by Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology was used to screen newborns exhibiting high-risk clinical phenotypes of genetic metabolic diseases from January 2023 to March 2024. Meanwhile, the clinical data were collected and follow-up observations were conducted to analyze the detection rates of pathogenic genes and the correlation between genotype and phenotype of the neonates. Results A total of 53 neonates with high-risk inherited metabolic clinical phenotypes were included in this study, among which the overall positive detection rate for pathogenic genes was 17.0% (9/53). The pathogenic genes detected in this study were chloride channel (CLCN1), dual oxidase 2 (DUOX2), gap junction protein beta 2 (GJB2), tyrosine-protein phosphatase nonreceptor type 11 (PTPN11), sodium channel α subunit (SCN1A), spastic paraplegia gene 11(SPG11), etc. DUOX2 and GJB2 were the most frequently detected, accounting for 33.3% (3/9) and 22.2% (2/9) of positive cases, respectively. A comparative analysis was made between the positive and negative groups of pathogenic genes, and it was found that the prognosis of the positive group was more serious and the number of clinical phenotypes might be more than that of the negative group. In the positive group of pathogenic genes, except for cases 1 and 9, whose clinical phenotypes were temporarily inconsistent with pathogenic genes due to the late onset of related diseases, the pathogenic genes were consistent with clinical phenotypes in the remaining cases. Conclusion The gene panel, characterized by short detection time, low detection cost and minimal technical demands, are suitable for the early screening of neonates with suspected genetic metabolic diseases in developing countries and economically backward areas.

Alport syndrome is a type of hereditary kidney disease caused by mutations in the type IV collagen gene. Depending on the genetic mode, it can be divided into X-linked Alport syndrome, autosomal dominant Alport syndrome, autosomal recessive Alport syndrome, and digenic Alport syndrome. The clinical manifestations are heterogeneous, ranging from isolated hematuria or hematuria with proteinuria to progressive renal failure, with or without extrarenal manifestations. This article reviews the genetic characteristics, biomarkers, and treatment-related research of Alport syndrome, aiming to provide reference for enhancing early diagnosis and treatment and optimize long-term prognosis.

Objective To report two cases of dyskeratosis congenita (DC) and provide a comprehensive literature review to improve the understanding of the disease. Methods Clinical characteristics of two DC cases were retrospectively collected and analyzed in Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology. Gene mutations were assessed by high-throughput sequencing analysis and telomere length was assessed by Terminal Restriction Fragment (TRF) analysis. A literature search was carried out using the National Knowledge Infrastructure (CNKI), Wanfang database, PubMed, and Web of Science, updated to June 2024, with “Dyskeratosis congenita” and “telomere biology disorders” as the keywords. Results Case 1 was a boy admitted with “nail dystrophy of fingers and toes for more than 8 years and pancytopenia for 1 week”. Physical examination revealed fingernails and toenails dysplasia, reticular skin pigmentation over the neck, and restricted mouth opening. Genetic testing identified a mutation in the DKC1 gene and shorter telomeres. Case 2 was a girl admitted with “confirmed aplastic anemia over 3 years”. Physical examination showed no specific abnormalities. A blood routine test showed pancytopenia, with missense mutations found in the RTEL1 and TERT genes. Case 1 received blood transfusion support, while Case 2 was treated with subcutaneous injections of PEGylated recombinant human granulocytes, cyclosporine, and eltrombopag olamine, but the outcomes were not satisfactory. Both cases developed bone marrow failure, prompting hematopoietic stem cell transplantation. However, both cases were lost to follow-up after discharge. Conclusions Dyskeratosis congenita is a rare disease with various clinical manifestations. It may present with skin manifestations or hematological abnormalities. A precise diagnosis is made through a genetic testing. Currently, efficacious medical treatment for DC is lacking, and hematopoietic stem cell transplantation is necessary for patients with bone marrow failure.

Objective This study aims to analyze the clinical and genetic characteristics of 14 children with neurofibromatosis type 1 (NF1) presenting with short stature, with the goal of enhancing clinical awareness and promoting early diagnosis and intervention. Methods Clinical data were collected from 14 children diagnosed with NF1 and short stature at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, between May 2017 and June 2024. The clinical characteristics, treatment approaches, and follow-up outcomes were analyzed. Results Among the 14 patients, there were 7 boys and 7 girls. The average age at diagnosis was 6.61±3.53 years old, with a height standard deviation score (SDS) of -2.60±0.66. All patients had scattered café-au-lait spots on their skin, with an averaging number of 9.64±4.86, and the largest diameter was 6 cm. Genetic testing identified five types of NF1 gene mutations: 6 nonsense mutations, 4 missense mutations, 2 frameshift mutations, 1 splice mutation, and 1 whole-gene mutation. During the follow-up, three patients developed cutaneous neurofibromas during puberty, four experienced an increase in the number of café-au-lait spots, and one developed tibial pseudarthrosis. Growth hormone (GH) stimulation tests were conducted on 10 patients. Two patients with GH deficiency received recombinant human growth hormone (rhGH) treatment, which resulted in a slightly increase in their growth rate. The remaining 12 children, who did not receive rhGH treatment, showed no significant change in growth rate during follow-up. Conclusion The clinical manifestations of NF1 are diverse and age-related. In children with short stature and multiple café-au-lait spots, NF1 should be considered as a potential diagnosis. Genetic testing should be conducted if necessary, and regular monitoring is essential to assess the disease’s evolution and to enable prompt interventional strategeies.

Objective To improve clinicians’ understanding of the diagnosis and treatment of neonatal hyperthyroidism which is rare in clinical practice. Methods The clinical data of a neonatal hyperthyroidism case diagnosed and treated was retrospectively analyzed, and the relevant literature was reviewed. Results A female infant, borned at 32 weeks and 6 days of gestation, exhibited dyspnea, a rapid heart rate, hepatosplenomegaly, thrombocytopenia, cholestasis, and abnormal liver function that continued to deteriorate after birth. She was later diagnosed with neonatal hyperthyroidism after undergoing thyroid function and antibody tests. After treated with methimazole, propranolol and other symptomatic supportive therapies, the infant’s symptoms improved and she was discharged in good health. Conclusions The clinical manifestations of neonatal hyperthyroidism are non-specific and the symptoms are atypical. Therefore, when encountering symptoms such as growth retardation, tachycardia, cardiac insufficiency, tachypnea, thrombocytopenia, liver injury, hepatosplenomegaly, and a small head circumference, it is essential to consider the possibility of congenital hyperthyroidism. Timely thyroid function and antibody tests should be conducted to confirm the diagnosis, followed by early drug intervention to enhance the prognosis.

Objective To explore the efficacy and safety of different antiplatelet drugs combined with rivaroxaban in patients with lower extremity arteriosclerosis obliterans. Methods The clinical data of patients with lower extremity arteriosclerosis obliterans who were symptomatic and underwent surgical treatment at the Vascular Surgery Department of Nanjing Drum Tower Hospital from January 2018 to December 2021 were retrospectively analyzed. According to the different antiplatelet medications taken by patients, patients were categorized into aspirin group, clopidogrel group and cilostazol group. Baseline data of patients were collected, and patients were followed up and the incidence of major adverse cardiovascular events, major adverse limb events, major bleeding and clinically related non-major bleeding events were compared in different groups. Results A total of 632 patients were included in the study. There was no significant difference in the incidence of major adverse cardiovascular events, major adverse limb events, major bleeding and clinically related non-major bleeding events after the baseline data was balanced by inverse probability of treatment weighting. The results of subgroup analysis were generally consistent with those of the overall study. Conclusion The combination of clopidogrel or cilostazol with rivaroxaban may serve as a novel option for dual antithrombotic therapy in patients diagnosed with lower extremity arteriosclerotic obliterans.

Heracleum candicans is extensively utilized for its abundant medicinal components, which are applied in various treatments, including insecticides, hemostatic agents, dispersing wind, relieving cough, and alleviating rheumatic pain. This paper reviewed the various chemical constituents and pharmacological effects of Heracleum candicans. It aims to provide new insights for its promotion and application, as well as to provide a basis for establishing quality standards for Heracleum candicans.

Objective This study aims to discuss the research hotspot and development trend in the field of polycystic ovary syndrome(PCOS)through bibliometric statistics and visual analysis of long noncoding RNA (lncRNA)related studies. Methods Utilizing the Web of Science core database as the literature data source, we searched for PCOS lncRNA-related literature from 2015 to 2023. CiteSpace software was used to conduct a visual analysis, including the annual distribution, citation trends, countries, institutions, funding sources and key words, as well as co-occurrence and cluster analysis of key words. Results The visual analysis of 108 PCOS lncRNA literature revealed that China was the country with the highest number of publications. The first contributing institution was the Shandong University. The national natural science fund of China gave the biggest funding. The keyword cluster analysis suggested that PCOS lncRNA signal pathway regulation, related receptor activators, and the expression of regulatory factors were the research hotspots in ovary syndrome lncRNA research. Conclusion LncRNA related regulatory factors, bioinformatics analysis, and gene transcription in PCOS are new targetsfor PCOS treatment, providing valuable insights for clinical therapy and new strategies for the development of PCOS-related pharmaceuticals.

Objective To establish a more scientific and reasonable evaluation method for the heat resistance, freezing resistance, and thermal stability of glass containers for pharmaceutical packaging to meet the regulatory needs of drugs in the new situation. Methods A survey was conducted on domestic injection production enterprises in the form of a questionnaire, including the process parameters of cleaning for glass containers for pharmaceutical packaging, terminal sterilization during injection production, and the freeze-drying process flow of freeze-dried formulations. Results The parameters of dry heat sterilization, freeze-drying, and moist heat sterilization used in actual production of injections vary widely, and evaluation through laboratory simulation under single conditions lacks scientific and practical applicability. Conclusions In order to implement the construction of the new standard system for glass packaging materials in 2025 edition of the Chinese Pharmacopoeia, through this investigation, the actual production process parameters of drugs were shown, and it is proposed that specific experimental conditions for heat resistance, freezing resistance, and thermal stability will no longer be limited in the new standard system for use. Suitable experimental conditions can be set by enterprises based on their actual process to evaluate performance.

Objective To prepare Zishen pills as gel plaster according to the prescription, and investigate its transdermal absorption characteristics in vitro. Methods Based on preliminary experiments, the matrix prescription of the gel plaster was optimized by single-factor tests and the Box-Behnken design. Evaluation indicators included initial viscosity, viscosity retention and sensory scores. The modified Franz diffusion cell was used to investigate the effect of penetration enhancers on the transdermal characteristics of gel plaster in vitro, with the permeability of neomangiferin, phellodendrine hydrochloride, mangiferin and berberine hydrochloride as evaluation indicators. Results The prescription dosage of the preferred matrix for the Zishen gel plaster was sodium polyacrylate NP700 2.55 g, glycerin 11.04 g, polyvinylpyrrolidone K90 1.13 g,tartaric acid 0.1 g, glycyrrhizin 0.1 g, kaolin 0.3 g, and distilled water 15 g. Among different types and concentrations of permeation enhancers, 5% aminoketone showed the best permeation performance. The permeation rates for neomangiferin, phellodendrine hydrochloride, mangiferin, and berberine hydrochloride were 1.5338, 1.7809, 2.3247 and 20.0899 μg·(cm²)^-1·h^-1,and the penetration rates were 2.4319, 1.9408, 1.9604 and 1.4701, respectively. The percutaneous absorption curve of the drug conformed to the zero-order kinetic equation. Conclusion The preparation process of the obtained gel plaste is stable and feasible, with good adhesive properties, sustained drug release, and favorable in vitro percutaneous permeability, indicating potential clinical application value.

Objective To evaluate the cost-effectiveness of roxadustat and darbepoetin alfa on treating renal anemia in dialysis-dependent chronic kidney disease (DD-CKD) patients, thus providing health economics reference for treatment of renal anemia. Methods A Markov model simulating the development and treatment of anemia in DD-CKD patients in a lifetime horizon (20 years) was constructed. Total costs of roxadustat and darbepoetin alfa injection were estimated from the perspective of Chinese healthcare system, with health outcomes converted into quality-adjusted life year (QALY). The incremental cost-effectiveness ratio (ICER) was used to describe the results. The willingness-to-pay (WTP) threshold was set at 257 094 yuan,which was three times China’s gross domestic product (GDP) per capita in 2023. Sensitivity analyses were performed to test the uncertainties of the results. Results The total treatment costs of roxadustat and darbepoetin alfa injection were 111 902.41 yuan and 52 927.92 yuan respectively, corresponding to QALY values of 4.76 and 4.74 life-years. The incremental cost-effectiveness (ICER) was 2 654 912.45 yuan/QALY, which exceeded 3 times GDP per capita. Therefore, compared with darbepoetin alfa injection, roxadustat has no cost-effectiveness for patients with DD-CKD. Conclusion In the context of current economic development in China, darbepoetin alfa injection is more cost-effective than roxadustatin for treating anemia in DD-CKD patients.

Objective To analyze the blood concentration monitoring results of 7 new antiepileptic drugs levetiracetam (LEV), oxcarbazepine (OXC), lamotrigine (LTG), topiramate (TPM), lacosamide (LCM), zonisamide (ZNS) and perampanel (PER) and provide a basis for clinical rational drug use. Methods Aretrospective analysis was conducted on the blood concentration monitoring results of 7 new antiepileptic drugs in a grade-A tertiary hospital in Beijing from November 2021 to March 2023, with a total of 6 537 valid concentration data collected.The patients were grouped according to age, gender and concomitant medication, and the blood drug concentration levels and compliance rates among the groups were analyzed and compared. Results The male to female patient ratio was 1.35:1. There were statistically significant differences in the blood concentration distribution of OXC, LEV, LCM and TPM between genders (P<0.05).The blood concentration of LEV showed statistically significant differences between the pediatric group and the elderly group, as well as between the young adult group and the elderly group (P<0.05).The blood concentrations of OXC, ZNS and TPM showed statistically significant differences between the pediatric group and the young and middle-aged group, between the young and middle-aged group and the elderly group, and between the pediatric group and the young and middle-aged group, respectively (P<0.05).The highest and lowest overall compliance rates of blood concentration were observed for OXC and LCM, respectively. The compliance rates of OXC and TPM in the pediatric group were significantly higher than those in the young-middle-aged group, with statistically significant differences (P<0.05), while the compliance rate of LEV in the elderly group was significantly higher than that in the pediatric group and the young-middle-aged group, with a statistically significant difference (P<0.05).There were a total of 2 133 cases with combined drug use. LEV, OXC and LTG are frequently used and have good efficacy and weak interactions when added to treatment. Conclusion New antiepileptic drugs show a promising prospect in treatment, and therapeutic drug monitoring can further improve the effectiveness of individualized clinical treatment.

Objective To investigate the compatible stability of esketamine hydrochloride, sufentanil citrate, butorphanol tartrate, and metoclopramide in 0.9% sodium chloride injection, and to provide a reference for the rational use of medication for postoperative analgesia. Methods The four drugs were compounded by simulating the clinical concentrations of the drugs, and the samples were taken at 0, 4, 8, 12, 24 and 48 h under light-shielded and light-exposed conditions at room temperature. The appearance, pH value, relative drug content, and insoluble particles were determined. Results The appearance of esketamine hydrochloride, sufentanil citrate, butorphanol tartrate, and metoclopramide concoction was clear at 48 h. The pH values were in the range of 4.69 to 4.79, and the relative drug content of the four drugs were in the range of 95% to 105%. The number of insoluble particles exceeded the specified range in the Chinese Pharmacopoeia (2020 edition) at 4 h and 8 h. Conclusions Under light-shielded and light-exposed conditions at room temperature, the appearance, pH value and relative drug content of the compounded solution remained stable for 48 h. However, the number of insoluble particles exceeded the specified standard. Therefore, it is not recommended to mix the above 4 drugs with 0.9% sodium chloride injection for use in analgesic pumps.

Objective To evaluate the efficacy, safety and economy of centrally procured ceftazidime injection for treating pulmonary infections, and to provide a reference for clinical therapeutic decisions. Methods Based on the active monitoring and drug evaluation functions of the China Hospital Pharmacovigilance System(CHPS), a retrospective analysis was conducted. Electronic medical records of 203 patients treated with ceftazidime injection for pulmonary injections in our hospital from February 2021 to August 2022 were collected. Patients were divided into two groups based on the type of ceftazidime used,the centrally procured ceftazidime group (102 cases) and the non-centrally procured ceftazidime group(101 cases). Efficacy was evaluated by the effectiveness rate, safety by the incidence of adverse reactions, and economy by cost-effectiveness analysis.Differences between the two groups were compared. Results Treatment efficiency was 90.20% in the centrally procured group and 91.09% in the non-centrally procured group,with no statistically significant difference (P>0.05). The incidence of adverse reactions was 10.78% in the centrally procured group and 5.94% in the non-centrally procured group, with no statistically significant difference (P>0.05). The average cost-effectiveness ratio (C/E) was 1.56±0.86 in the centrally procured group and 18.96±9.38 in the non-centrally procured group, indicating a smaller C/E value for the centrally procured group. Sensitivity analysis results were consistent with the cost-effectiveness analysis results. Conclusion Centrally procured ceftazidime injection has equivalent efficacy and safety compared to non-centrally procured ceftazidime, with improved economic value. Therefore, the centrally procured variant should be given priority when using ceftazidime for the treatment of pulmonary infections.

Objective To analyze the diagnosis and treatment process of a pediatric patient with Crohn's disease (CD) who was treated with multiple biological agents, and to provide a reference for the treatment and pharmaceutical care. Methods The diagnosis and treatment process of the pediatric patient’s disease was analyzed. Drug treatment plans were evaluated through literature research to identify the entry points for pharmaceutical care and medication guidance. Results Clinical pharmacist monitored the pediatric patient's adalimumab maintenance dose, efficacy, and adverse reactions through therapeutic drug monitoring. They recommended clinical adjustments to the maintenance dose of biological agents, so that the pediatric patients could benefit from multiple aspects such as efficacy, safety, and economy. Conclusion Clinical pharmacists should follow up with pediatric patients with CD, and pay attention to medication compliance and adverse reactions in those using biological agents.

Objective To propose strategies for developing clinical predictive models, aiming to assist researchers in conducting standardized clinical prediction model studies. Methods Literature review was conducted to summarize the operational steps and content for developing clinical predictive models. Then, a methodological framework was summarized and refined through expert consultation. Results The 11-step methodological framework for developing clinical predictive models was obtained by synthesizing the experience of 456 clinical predictive modeling studies and expert consultation, and the details were analyzed and elaborated. Conclusions This study presents methodological strategies and recommendations for the development of clinical predictive models, intended to serve as a guide for researchers.

This paper summarizes the policy background of the promotion and application of electronic certificates, introduces the sharing service model of electronic certificates, summarizes the current status of the application of electronic certificates in the field of drug regulation, analyzes the difficulties and problems of electronic certificate application from aspects such as management methods and service guides, mutual recognition mechanisms, certificate standards and specifications, application convenience, and the technology of fixed layout document, and puts forward countermeasures and suggestions.

Objective This study aims to assess the current status of pharmaceutical management in county medical communities in Hubei province, and provide recommendations for the homogenization, standardization, and regulation of pharmaceutical management in these communities. It also intends to offer decision-making support for health administrative departments, and provide reference experiences for management in other regions. Methods The current status of pharmaceutical management in county medical communities in Hubei province was conducted through a questionnaire survey and field research. Existing problems were analyzed, key management areas were identified, and reasonable recommendations were proposed. Results Pharmaceutical management in county medical communities has significant shortcomings in organizational structure, system construction, personnel allocation, key link control, and the leading unit's outreach capabilities. These deficiencies are not aligned with the high-quality development of pharmacy in the new era. Conclusions It is recommended that county medical communities should establish a comprehensive pharmaceutical management quality control system. This can be achieved by improving organizational management, strengthening talent development, enhancing core systems, setting monitoring indicators, and increasing outreach capabilities. Additionally, evaluation standards for the quality control system of pharmaceutical management should be established to enhance management capabilities through scientific assessment and positive feedback.